Autosomal recessive cerebellar ataxia
MONDO:0015244Autosomal recessive cerebellar ataxias (ARCA) are a heterogeneous group of rare neurological disorders involving both the central and peripheral nervous system (and in some cases other systems and organs), and characterized by degeneration or abnormal development of the cerebellum and spinal cord and, in most cases, early onset occurring before the age of 20 years.
Also known as: ARCA, arca, cerebellar ataxia, autosomal recessive
90 clinical trials for this condition and its sub-types, 0 tagged with Autosomal recessive cerebellar ataxia itself.
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Browse by category →Sub-types of Autosomal recessive cerebellar ataxia
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Autosomal recessive degenerative and progressive cerebellar ataxia 0 trials · 49 incl. sub-types
7 sub-types
- Friedreich ataxia 37 trials Sub-types →
- Marinesco-Sjogren syndrome 10 trials
- FLVCR1-related retinopathy with or without ataxia 0 trials · 1 incl. sub-types Sub-types →
- Mitochondrial DNA depletion syndrome 7 (hepatocerebral type) 1 trial
- Congenital cataracts-facial dysmorphism-neuropathy syndrome 0 trials
- Early-onset cerebellar ataxia with retained tendon reflexes 0 trials
- Early-onset progressive encephalopathy-spastic ataxia-distal spinal muscular atrophy syndrome 0 trials
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Autosomal recessive metabolic cerebellar ataxia 0 trials · 10 incl. sub-types
7 sub-types
- Cerebrotendinous xanthomatosis 6 trials
- Abetalipoproteinemia 2 trials
- Recessive mitochondrial ataxia syndrome 2 trials
- Familial isolated deficiency of vitamin E 1 trial
- Autosomal recessive ataxia due to PEX10 deficiency 0 trials
- Autosomal recessive cerebellar ataxia - pyramidal signs - nystagmus - oculomotor apraxia syndrome 0 trials Sub-types →
- Autosomal recessive cerebellar ataxia with late-onset spasticity 0 trials
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Autosomal recessive congenital cerebellar ataxia 0 trials · 7 incl. sub-types
7 sub-types
- Cerebellar ataxia, intellectual disability, and dysequilibrium 5 trials Sub-types →
- Joubert syndrome and related disorders 0 trials · 2 incl. sub-types Sub-types →
- CAMOS syndrome 0 trials
- Cayman type cerebellar ataxia 0 trials
- Autosomal recessive spinocerebellar ataxia 17 0 trials
- Autosomal recessive spinocerebellar ataxia 2 0 trials
- Congenital cerebellar ataxia due to RNU12 mutation 0 trials
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Autosomal recessive syndromic cerebellar ataxia 0 trials · 2 incl. sub-types
7 sub-types
- Cerebellar ataxia with neuropathy and bilateral vestibular areflexia syndrome 2 trials
- Gemignani syndrome 0 trials
- Acute infantile liver failure-cerebellar ataxia-peripheral sensory motor neuropathy syndrome 0 trials
- Ataxia - oculomotor apraxia type 4 0 trials
- Autosomal recessive cerebellar ataxia-saccadic intrusion syndrome 0 trials
- Autosomal recessive spinocerebellar ataxia 11 0 trials
- Peroxisome biogenesis disorder 4B 0 trials
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Spinocerebellar ataxia, autosomal recessive, with axonal neuropathy 0 trials · 2 incl. sub-types
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Lichtenstein-Knorr syndrome 0 trials
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RIDDLE syndrome 0 trials
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3 sub-types
Most studied deeper sub-types
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Experimental Friedreich's ataxia drug tested in kids – but trial halted early
Disease control Stopped earlyThis early-stage trial tested a drug called nomlabofusp (CTI-1601) in 18 adolescents and children with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to check safety and how the body processes the drug. However, the study was term…
Phase 1 • Sponsor: Larimar Therapeutics, Inc. • Aim: Disease control
Last updated Jul 12, 2026 00:00 UTC
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Virus therapy fails to advance in colorectal cancer trial
Disease control Stopped earlyThis study tested a new approach using a virus that attacks cancer cells (oncolytic immunotherapy) along with two standard drugs (atezolizumab and bevacizumab) in people with advanced colorectal cancer that had stopped responding to other treatments. The trial was stopped early a…
Phase 2 • Sponsor: Replimune, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:01 UTC
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Experimental cell therapy targets deadly childhood brain cancer
Disease control Stopped earlyThis early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…
Phase 1 • Sponsor: University of Florida • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC